ATL1103 Phase II Trial Results Published in the European Journal of Endocrinology

MELBOURNE, Australia, May 23, 2018 (Newswire.com) - Antisense Therapeutics (the “Company” or “ANP”) is pleased to announce the acceptance for publication of previously reported positive Phase II clinical trial data on ATL1103 (atesidorsen) in the leading peer-reviewed medical journal, the European Journal of Endocrinology.

An abstract of the article titled "A randomised, open-label, parallel group phase 2 study of antisense oligonucleotide therapy in acromegaly", is currently available online http://dx.doi.org/10.1530/EJE-18-0138, with the full manuscript to be included in a future print edition and anticipated to be made available free on line in due course.

The article highlights the successful outcomes of the Phase II clinical trial of ATL1103 in acromegaly patients where the safety, tolerability, pharmacokinetics, and efficacy of two subcutaneous dosing regimens of ATL1103 in 26 adult acromegaly patients dosed with ATL1103 for 13 weeks were assessed. ATL1103 met its primary endpoint in the study resulting in a median fall in serum insulin-like growth factor-I (sIGF-I) of 27.8% (p=0.0002) at the twice-weekly 200mg dose tested.

The authors of the publication include the clinical investigators from the Phase II study who are prominent endocrinologists from centres in the UK, France, Spain, and Australia. The Principal Investigator of the ATL1103 Phase II study, Dr Peter Trainer, Professor of Endocrinology, The Christie NHS Foundation Trust, UK, is the lead author of the publication.

Antisense Therapeutics CEO and Managing Director Mark Diamond said:

”The European Journal of Endocrinology has a global readership of over 70,000 unique users worldwide who access the journal online each month and so we are delighted to have our ATL1103 Phase II data published in a high-quality scientific journal that has such a broad reach to the target audience for our acromegaly program. The publication is expected to be very beneficial for the Company’s plans to conduct an ATL1103 Early Access Program in Europe and for future initiatives with the drug and also provides important independent validation to the quality of our data and the benefits that ATL1103 may bring to the lives of patients with acromegaly.”

Contact Information:

Website: www.antisense.com.au

Email:  [email protected]

Managing Director: Mark Diamond +61 (0)3 9827 8999

About Antisense Therapeutics Limited (ASX: ANP) is an Australian publicly listed biopharmaceutical company, developing and commercialising antisense pharmaceuticals for large unmet markets. The products are in-licensed from Ionis Pharmaceuticals Inc. world leaders in antisense drug development and commercialisation. ATL1102 (injection) has successfully completed a Phase II efficacy and safety trial, significantly reducing the number of brain lesions in patients with relapsing-remitting multiple sclerosis (RRMS). ATL1103 drug designed to block GHr production successfully reduced blood IGF-I levels in Phase II clinical trials in patients with the growth disorder acromegaly. The Company is planning to conduct a Phase II clinical trial of ATL1102 in DMD patients at the Royal Childrens Hospital, Melbourne.

About Early Access Programs, Early Access Programs allow biopharmaceutical companies to provide eligible patients with ethical access to investigational medicines for unmet medical needs within the scope of the existing early access legislation.  Access is provided in response to physician requests where other treatments have been unsuccessful and no alternative or appropriate treatment options are available to these patients. In Europe, the Company can seek reimbursement of drug supply costs within select countries. In Australia, patient treatment via an Early Access Program would typically fall under the Therapeutic Goods Administration’s Special Access Scheme, Personal Import Scheme or Authorised Prescribers pathway.

About Acromegaly Acromegaly is a serious chronic life-threatening disease triggered by excess secretion of growth hormone (GH) by a benign tumour of the pituitary. Oversupply of GH produces excess levels of Insulin-Like Growth Factor-I (IGF-I) in the blood causing the abnormal growth of the bones of the face, hands and feet, and enlargement of body organs. In North America and Europe, there are approximately 85,000 acromegaly patients with around one half of these requiring life-long drug therapy.  A significant number of patients fail to be adequately treated with current medicines due to efficacy, safety or tolerance related issues. The current average cost for 2nd line acromegaly treatment in Europe is approximately A$80K per patient per annum.

About ATL1103 / atesidorsen ATL1103 is a second-generation antisense drug designed to block growth hormone receptor (GHr) expression thereby reducing levels of the hormone insulin-like growth factor-1 (IGF-1) in the blood and is a potential treatment for diseases associated with excessive growth hormone and IGF-1 action. These diseases include acromegaly, an abnormal growth disorder of organs, face, hands and feet, diabetic retinopathy, a common disease of the eye and a major cause of blindness, diabetic nephropathy, a common disease of the kidney and major cause of kidney failure, and some forms of cancer. Acromegalic patients have significantly higher blood IGF-1 levels than healthy individuals. Reduction of these levels to normal is accepted by clinical authorities as the primary marker of an effective drug treatment for the disease. GHr is a clinically validated target in the treatment of acromegaly. In the case of diabetic retinopathy, published clinical studies have shown that treatments producing a reduction in IGF-1 levels retarded the progression of the disease and improve vision in patients. Scientific papers have been published on the suppression of blood IGF-1 levels in mice (Tachas et al., 2006, J Endocrinol 189, 147-54) and inhibition of retinopathy in a mouse retinopathy model (Wilkinson-Berka et al., 2007, Molecular Vision 13, 1529- 38) using an antisense drug to inhibit the production of GHr. In a Phase I study in healthy subjects, ATL1103 demonstrated a preliminary indication of drug activity, including suppression of IGF-1 and the target GHr (via circulating growth hormone binding protein) levels. In a Phase II trial in acromegalic patients, ATL1103 met its primary efficacy endpoint by showing a statistically significant average reduction in sIGF-1 levels from baseline at week 14 (one week past the last dose) at the twice-weekly 200 mg dose tested. Antisense has also completed a successful higher dose study in acromegaly patients. ATL1103 has Orphan Drug designation in the US and Europe.

Source: Antisense Therapeutics Limited